Dear CTNNB1 community,
This month marks a truly extraordinary moment in our shared journey – the approval of our first-ever clinical trial for CTNNB1 syndrome. Together, we’ve turned what once felt like a distant dream into a concrete reality.
Every late night, every donation, every show of support has led us to this point.
In this edition, we celebrate this groundbreaking milestone, share exciting updates about the Dragonfly Study’s expansion to Australia, announce the 2026 International CTNNB1 Conference in Barcelona, and highlight our continued fundraising efforts to reach the final stretch toward treatment.
We also bring you an inspiring story of hope – Edan’s journey with gene therapy for SMA, a reminder of the life-changing potential that lies ahead for CTNNB1 children.
👉 Download the November 2025 Newsletter (PDF)
Best,
The CTNNB1 Team